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PENDLRA trial of low-dose sirolimus for Pendred syndrome/DFNB4
jRCT2091220361Phase 1Completed
- Sponsor
- Keio University
- Start
- Jul 2018
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 1, randomised, double-blind trial in Pendred syndrome (PDS) and deafness comparing NPC-12T with Placebo (2 arms). Primary endpoint: Incidence and grade of adverse events and adverse reactions. Started Jul 2018; status: completed.
02
Drugs
sirolimus
03
Conditions
Pendred syndrome (PDS)deafness
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